相关临床试验
112
15 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
1997
进行中（未招募）
12
10.7%
Approved For Marketing
3
2.7%
已完成
60
53.6%
Enrolling By Invitation
3
2.7%
No Longer Available
1
0.9%
招募中
12
10.7%
终止
19
17.0%
Unknown
1
0.9%
撤回
1
0.9%
暂无批准数据
- BioMarin's Phase 3 CANOPY-HCH-3 trial met its primary endpoint, with VOXZOGO (vosoritide) improving annualized growth velocity by 2.33 cm/year versus placebo after 52 weeks (p<0.0001). - The study enrolled 81 children aged 3 to 17 with genetically confirmed hypochondroplasia and also showed significant gains in standing height, height Z-score and arm span. - BioMarin has submitted a supplemental New Drug Application to the FDA seeking the first approved targeted therapy for hypochondroplasia, with a potential 2027 launch. - Safety findings were consistent with VOXZOGO's established profile in achondroplasia, with mostly mild adverse events and no treatment-related serious adverse events.
- BioMarin Pharmaceutical will acquire privately held Alesta Therapeutics for $275 million up front, with up to $215 million more in development and regulatory milestones. - Alesta's lead candidate ALE1 is in a Phase 1/2 trial for hypophosphatasia, a genetic disease affecting bone mineralization that can cause fractures, tooth loss, and muscle weakness. - ALE1 acts on a novel target that lowers inorganic pyrophosphate, a metabolite central to hypophosphatasia, and could reach BioMarin's largest addressable patient population. - The deal, BioMarin's third acquisition in two years, is expected to close by the end of September and will be funded with cash on hand.
- The National Organization for Rare Disorders (NORD) has appointed Kathryn Lowell as Executive Vice President of Government Affairs, effective June 9, 2026. - Lowell brings 15 years of experience from BioMarin Pharmaceutical, where she most recently served as Group Vice President of Global Government Affairs & Advocacy. - In her new role, Lowell will lead policy initiatives aimed at accelerating R&D, improving regulatory and reimbursement pathways, and expanding access to rare disease therapies. - NORD represents over 30 million Americans living with rare diseases and partners with more than 350 disease-specific patient advocacy organizations.
- Children with five genetic subtypes of short stature experienced significant growth acceleration from 4.53 cm/year to 8.09 cm/year during vosoritide treatment in a phase 2 basket trial. - The study represents the first precision medicine approach for MAPK pathway growth disorders, with NPR2 deficiency patients showing particularly robust responses without significant adverse events. - Long-term safety concerns emerged as five participants discontinued treatment due to orthopedic complications including slipped capital femoral epiphyses and severe genu valgum, particularly in children with ACAN mutations. - The trial demonstrates potential for expanding vosoritide use beyond its current achondroplasia indication, though careful monitoring is essential for certain genetic subtypes.
- Over 180 companies are advancing 250+ AAV-based gene therapy candidates globally, spanning preclinical through marketed stages, according to DelveInsight's 2026 pipeline assessment. - The FDA granted approval to Otarmeni, the first dual-AAV gene therapy for OTOF-related genetic hearing loss, and accepted Ultragenyx's BLA for UX111 in Sanfilippo syndrome Type A with a September 2026 PDUFA date. - Taysha Gene Therapies reported FDA alignment on a BLA pathway for TSHA-102 in Rett syndrome, with pivotal REVEAL trial enrollment progressing and manufacturing validation underway. - Sensorion's SENS-501 demonstrated durable efficacy and favorable safety at six months in a Phase I/II trial for otoferlin-related congenital hearing loss, while GJB2-GT advances toward regulatory filing.
- Absci Corporation successfully dosed the first three cohorts in the single ascending dose portion of its Phase 1/2a HEADLINE trial for ABS-201, with the anti-PRLR antibody showing favorable emerging safety data and good tolerability. - Human ex vivo studies demonstrated that ABS-201 effectively stimulates hair growth by regenerating the stem cell niche and significantly inhibiting the PRLR signaling pathway, correlating with prolonged anagen phase and restoration of growth signaling. - The company plans to report interim proof-of-concept data for androgenetic alopecia in the second half of 2026 and initiate a Phase 2 trial for endometriosis by year-end, targeting two conditions with significant unmet medical needs.
- The FDA has approved BioMarin's supplemental Biologics License Application for PALYNZIQ (pegvaliase-pqpz) to include adolescents aged 12 and older with phenylketonuria (PKU). - The approval is based on the Phase 3 PEGASUS study, which demonstrated statistically significant blood phenylalanine reduction compared to diet alone in adolescents with uncontrolled PKU. - PALYNZIQ is the only enzyme substitution therapy approved for PKU treatment, offering a genotype-independent medication that may normalize phenylalanine levels while allowing unrestricted diet. - Nearly half of study participants (44.4%) achieved blood phenylalanine levels below guideline recommendations, with some able to significantly increase protein intake and reduce medical food dependency.
- BioMarin Pharmaceutical completed its first-ever senior notes offering, raising $850 million through 5.500% senior notes due 2034 to support strategic expansion. - The proceeds will finance BioMarin's acquisition of Amicus Therapeutics and debt repayment, combining two rare disease-focused biotechnology companies. - This transaction represents a significant consolidation move in the rare disease therapeutics sector, bringing together over 25 years of BioMarin's pioneering experience with Amicus's complementary portfolio.
- BridgeBio's oral drug infigratinib demonstrated superior growth acceleration in children with achondroplasia, achieving 1.74-2.1 cm per year improvement over placebo in a pivotal trial of over 100 patients. - The drug showed best-in-indication efficacy compared to existing injectable treatments, with BioMarin's Voxzogo showing 1.57 cm per year and Ascendis' therapy showing 1.49-1.78 cm per year in their respective trials. - Infigratinib was well-tolerated with no serious adverse events or treatment discontinuations due to side effects, positioning it as a convenient oral alternative to current daily injection therapies. - BridgeBio plans to submit marketing applications in the second half of 2026 and accelerate development in hypochondroplasia, potentially capturing significant market share in the growing dwarfism treatment space.
- Mendra launched with an $82 million Series A financing co-led by OrbiMed, 8VC, and 5AM Ventures to acquire and develop rare disease therapies using artificial intelligence. - The company plans to use AI to address key challenges in rare disease development including patient identification, clinical trial enrollment, and global market access planning. - Mendra's leadership team brings extensive experience from BioMarin Pharmaceutical and other rare disease companies, focusing on asset acquisition rather than drug discovery. - The funding reflects continued investor confidence in rare disease programs designed to address structural inefficiencies that limit progress beyond early development.
